Vaccine Wins FDA Approval for Advanced Prostate Cancer

The Food and Drug Administration has approved sipuleucel-T for treatment of advanced prostate cancer in a much-anticipated ruling that marks the first approval of a vaccine for cancer treatment.

The indication is for use in patients with “asymptomatic or minimally symptomatic prostate cancer that has spread to other parts of the body and is resistant to standard hormone treatment,” according to the FDA announcement. Sipuleucel-T will be marketed as Provenge by manufacturer Dendreon.

The company announced the vaccine will be available initially at 50 oncology and urology centers that were approved clinical trial sites. Executives said in an investors' call that they expected to treat the first patient within a week, and aim to serve 2,000 patients within the first 12 months. Initially, the individually tailored vaccine will be manufactured only in the company's New Jersey facility, but Dendreon plans to add facilities in Atlanta and in Orange County, Calif., by mid-2011.

Pricing has been set at $31,000 per infusion, or a total of $93,000 for the therapy. Executives said they plan to meet with Medicare officials about reimbursement; about three-fourths of the target population is Medicare eligible. The company also has set up a patient-access program to help men who cannot afford copayments.

The granting of the indication follows a long and tumultuous review process in which protestors picketed after an FDA advisory committee rejected Dendreon's initial application for the vaccine. Early results from a key trial designed to address issues raised by the panel failed to show an improvement in progression-free survival, but researchers were eventually able to demonstrate that men lived longer when treated with sipuleucel-T.

The pivotal Dendreon-sponsored, phase III IMPACT (Immunotherapy for Prostate Adenocarcinoma Treatment) trial randomized 512 men with metastatic castration-resistant prostate cancer to sipuleucel-T or placebo.

At a median follow-up of 3 years, the vaccine was credited with a 4.1-month gain in overall survival, with men on the vaccine living a median of 25.8 months vs. 21.7 months in the control group.

Adverse events occurred in almost all patients, with chills, fatigue, fever, back pain, nausea, joint ache, and headache being common reactions. Most side effects were mild or moderate, but the FDA noted that about a quarter of patients had serious adverse reactions, including some acute infusion reactions and stroke.

“Cerebrovascular events, including hemorrhagic and ischemic strokes, were observed in 3.5% of patients in the Provenge group, compared with 2.6% of patients in the control group,” the agency said.

The company announced that it has committed to conducting “a registry of approximately 1,500 patients to further evaluate a small potential safety signal of cerebrovascular events.”

An autologous cellular immunotherapy, sipuleucel-T delivers a patient's own immune cells, extracted via leukapheresis, in a vaccine designed to stimulate an immune response against prostatic acid phosphatase (PAP), an antigen expressed in most prostate cancers. Men received three doses of the vaccine in intravenous injections given at about 2-week intervals.

“The availability of Provenge provides a new treatment option for men with advanced prostate cancer, who currently have limited effective therapies available,” said Dr. Karen Midthun, acting director of the FDA's Center for Biologics Evaluation and Research, in the FDA announcement.

Emily Hayes of The Pink Sheet contributed to this report. The Pink Sheet and this publication are owned by Elsevier.

My Take

Price Will Be an Issue for Provenge

After a series of ups and downs, the much-debated prostate cancer vaccine Provenge is now FDA approved. Instead of the end of the discussion, the story is likely only beginning. The price is said to be in the $90,000 range for the treatment course (three monthly injections), and that number will further stimulate talk around “how much is it worth, for how long?” and who should receive the therapy.

With other high-priced therapies, such as bevacizumab (Avastin) or cetuximab (Erbitux), the doses are delivered repeatedly, and the overall cost only increases for those patients who are benefiting clinically. In the case of Provenge, the price is basically one size fits all, and there are no predictive tests as to which patients will likely benefit from the vaccine. Coupling the financial concerns with Dendreon's publicly stated manufacturing shortfall (which will limit access), the launch and utilization of this new therapy will be a story to follow closely in this era of health reform.

HOWARD A. BURRIS III, M.D., is chief medical officer and director of drug development at Sarah Cannon Research Institute in Nashville, Tenn., and editor of

The Food and Drug Administration has approved sipuleucel-T for treatment of advanced prostate cancer in a much-anticipated ruling that marks the first approval of a vaccine for cancer treatment.

The indication is for use in patients with “asymptomatic or minimally symptomatic prostate cancer that has spread to other parts of the body and is resistant to standard hormone treatment,” according to the FDA announcement. Sipuleucel-T will be marketed as Provenge by manufacturer Dendreon.

The company announced the vaccine will be available initially at 50 oncology and urology centers that were approved clinical trial sites. Executives said in an investors' call that they expected to treat the first patient within a week, and aim to serve 2,000 patients within the first 12 months. Initially, the individually tailored vaccine will be manufactured only in the company's New Jersey facility, but Dendreon plans to add facilities in Atlanta and in Orange County, Calif., by mid-2011.

Pricing has been set at $31,000 per infusion, or a total of $93,000 for the therapy. Executives said they plan to meet with Medicare officials about reimbursement; about three-fourths of the target population is Medicare eligible. The company also has set up a patient-access program to help men who cannot afford copayments.

The granting of the indication follows a long and tumultuous review process in which protestors picketed after an FDA advisory committee rejected Dendreon's initial application for the vaccine. Early results from a key trial designed to address issues raised by the panel failed to show an improvement in progression-free survival, but researchers were eventually able to demonstrate that men lived longer when treated with sipuleucel-T.

The pivotal Dendreon-sponsored, phase III IMPACT (Immunotherapy for Prostate Adenocarcinoma Treatment) trial randomized 512 men with metastatic castration-resistant prostate cancer to sipuleucel-T or placebo.

At a median follow-up of 3 years, the vaccine was credited with a 4.1-month gain in overall survival, with men on the vaccine living a median of 25.8 months vs. 21.7 months in the control group.

Adverse events occurred in almost all patients, with chills, fatigue, fever, back pain, nausea, joint ache, and headache being common reactions. Most side effects were mild or moderate, but the FDA noted that about a quarter of patients had serious adverse reactions, including some acute infusion reactions and stroke.

“Cerebrovascular events, including hemorrhagic and ischemic strokes, were observed in 3.5% of patients in the Provenge group, compared with 2.6% of patients in the control group,” the agency said.

The company announced that it has committed to conducting “a registry of approximately 1,500 patients to further evaluate a small potential safety signal of cerebrovascular events.”

An autologous cellular immunotherapy, sipuleucel-T delivers a patient's own immune cells, extracted via leukapheresis, in a vaccine designed to stimulate an immune response against prostatic acid phosphatase (PAP), an antigen expressed in most prostate cancers. Men received three doses of the vaccine in intravenous injections given at about 2-week intervals.

“The availability of Provenge provides a new treatment option for men with advanced prostate cancer, who currently have limited effective therapies available,” said Dr. Karen Midthun, acting director of the FDA's Center for Biologics Evaluation and Research, in the FDA announcement.

Emily Hayes of The Pink Sheet contributed to this report. The Pink Sheet and this publication are owned by Elsevier.

My Take

Price Will Be an Issue for Provenge

After a series of ups and downs, the much-debated prostate cancer vaccine Provenge is now FDA approved. Instead of the end of the discussion, the story is likely only beginning. The price is said to be in the $90,000 range for the treatment course (three monthly injections), and that number will further stimulate talk around “how much is it worth, for how long?” and who should receive the therapy.

With other high-priced therapies, such as bevacizumab (Avastin) or cetuximab (Erbitux), the doses are delivered repeatedly, and the overall cost only increases for those patients who are benefiting clinically. In the case of Provenge, the price is basically one size fits all, and there are no predictive tests as to which patients will likely benefit from the vaccine. Coupling the financial concerns with Dendreon's publicly stated manufacturing shortfall (which will limit access), the launch and utilization of this new therapy will be a story to follow closely in this era of health reform.

HOWARD A. BURRIS III, M.D., is chief medical officer and director of drug development at Sarah Cannon Research Institute in Nashville, Tenn., and editor of

The Food and Drug Administration has approved sipuleucel-T for treatment of advanced prostate cancer in a much-anticipated ruling that marks the first approval of a vaccine for cancer treatment.

The indication is for use in patients with “asymptomatic or minimally symptomatic prostate cancer that has spread to other parts of the body and is resistant to standard hormone treatment,” according to the FDA announcement. Sipuleucel-T will be marketed as Provenge by manufacturer Dendreon.

The company announced the vaccine will be available initially at 50 oncology and urology centers that were approved clinical trial sites. Executives said in an investors' call that they expected to treat the first patient within a week, and aim to serve 2,000 patients within the first 12 months. Initially, the individually tailored vaccine will be manufactured only in the company's New Jersey facility, but Dendreon plans to add facilities in Atlanta and in Orange County, Calif., by mid-2011.

Pricing has been set at $31,000 per infusion, or a total of $93,000 for the therapy. Executives said they plan to meet with Medicare officials about reimbursement; about three-fourths of the target population is Medicare eligible. The company also has set up a patient-access program to help men who cannot afford copayments.

The granting of the indication follows a long and tumultuous review process in which protestors picketed after an FDA advisory committee rejected Dendreon's initial application for the vaccine. Early results from a key trial designed to address issues raised by the panel failed to show an improvement in progression-free survival, but researchers were eventually able to demonstrate that men lived longer when treated with sipuleucel-T.

The pivotal Dendreon-sponsored, phase III IMPACT (Immunotherapy for Prostate Adenocarcinoma Treatment) trial randomized 512 men with metastatic castration-resistant prostate cancer to sipuleucel-T or placebo.

At a median follow-up of 3 years, the vaccine was credited with a 4.1-month gain in overall survival, with men on the vaccine living a median of 25.8 months vs. 21.7 months in the control group.

Adverse events occurred in almost all patients, with chills, fatigue, fever, back pain, nausea, joint ache, and headache being common reactions. Most side effects were mild or moderate, but the FDA noted that about a quarter of patients had serious adverse reactions, including some acute infusion reactions and stroke.

“Cerebrovascular events, including hemorrhagic and ischemic strokes, were observed in 3.5% of patients in the Provenge group, compared with 2.6% of patients in the control group,” the agency said.

The company announced that it has committed to conducting “a registry of approximately 1,500 patients to further evaluate a small potential safety signal of cerebrovascular events.”

An autologous cellular immunotherapy, sipuleucel-T delivers a patient's own immune cells, extracted via leukapheresis, in a vaccine designed to stimulate an immune response against prostatic acid phosphatase (PAP), an antigen expressed in most prostate cancers. Men received three doses of the vaccine in intravenous injections given at about 2-week intervals.

“The availability of Provenge provides a new treatment option for men with advanced prostate cancer, who currently have limited effective therapies available,” said Dr. Karen Midthun, acting director of the FDA's Center for Biologics Evaluation and Research, in the FDA announcement.

Emily Hayes of The Pink Sheet contributed to this report. The Pink Sheet and this publication are owned by Elsevier.

My Take

Price Will Be an Issue for Provenge

After a series of ups and downs, the much-debated prostate cancer vaccine Provenge is now FDA approved. Instead of the end of the discussion, the story is likely only beginning. The price is said to be in the $90,000 range for the treatment course (three monthly injections), and that number will further stimulate talk around “how much is it worth, for how long?” and who should receive the therapy.

With other high-priced therapies, such as bevacizumab (Avastin) or cetuximab (Erbitux), the doses are delivered repeatedly, and the overall cost only increases for those patients who are benefiting clinically. In the case of Provenge, the price is basically one size fits all, and there are no predictive tests as to which patients will likely benefit from the vaccine. Coupling the financial concerns with Dendreon's publicly stated manufacturing shortfall (which will limit access), the launch and utilization of this new therapy will be a story to follow closely in this era of health reform.

HOWARD A. BURRIS III, M.D., is chief medical officer and director of drug development at Sarah Cannon Research Institute in Nashville, Tenn., and editor of